Clinical Trial: Nordic Study on the Effects of Growth Hormone (Norditropin SimpleXx) Treatment in Adults With Prader-Willi Syndrome

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Nordic Study on the Effects of Growth Hormone (Norditropin SimpleXx) Treatment in Adults With Prader-Willi Syndrome

Brief Summary: The aim of this study is to study the effects of GH on body composition, lipid and glucose metabolism, physical performance and safety aspects in adults with PWS.The patients are randomized to either GH or placebo the first year of the study, subsequently followed by two years of GH treatment. the study is performed in Norway, Sweden and Denmark.

Detailed Summary:

OBJECTIVE(S):

Prader Willi syndrome (PWS) is a multi-symptomatic genetic disorder associated with abnormalities in the growth hormone (GH)-insulin-like-growth factor (IGF)-I axis and in the body composition. GH treatment is a registered indication in children with PWS, and improves growth rate and body composition. One pilot study in adult patients with clinical PWS has shown beneficial effects on body composition without simultaneous significant side effects. The aim of the present study is to evaluate the effects of GH treatment on body composition, muscle function and quality of life in PWS adults.

TRIAL DESIGN:

The study will be an investigator initiated and investigator sponsored multinational and multi-centre trial, including centres in Norway, Sweden and Denmark. Within each centre patients will be randomised (double blind) to one year treatment with daily injections of GH or placebo (efficacy), followed by a two year observation period on GH treatment (safety).

TRIAL POPULATION:

Twenty patients from each centre are included in the study. The patients need a genetically verified diagnosis and should be between 18 and 40 years old. Patients are excluded if GH treatment has been given within the last two years, if they have a malignancy or other serious diseases, in particular severe respiratory diseases.

ASSESSMENTS:

Effect is evaluated primarily as changes in body composition, activity of daily living and quality of life.

SAFETY: Before starting in the study all patients will be examined for tonsillary hypertrophy and sle
Sponsor: Karolinska University Hospital

Current Primary Outcome: Changes in body composition (lean body mass and fat mass) measured by dual energy X-ray absorptiometry (DXA) [ Time Frame: 36 months ]

Original Primary Outcome:

  • Changes in body composition (lean body mass and fat mass) measured by dual energy X-ray absorptiometry (DXA)
  • Muscle and fat mass measured by abdominal and mid-femoral computerized tomography (CT) scan
  • Activity of daily living measured a.m. Guralnik
  • Quality of life estimated by questionnaires


Current Secondary Outcome:

  • Bone mineral density measured by DXA [ Time Frame: 36 months ]
  • Effects on forced expiratory volume (Peakflow) [ Time Frame: 36 months ]
  • Standard photography appearance according to visual analogue scale (VAS) [ Time Frame: 36 months ]
  • Effects on free and total IGF-I, IGF-binding protein (BP)-1 and 3 [ Time Frame: 36 months ]
  • Effects on lipids (fasting triglycerides(TG), total, HDL and LDL cholesterol) [ Time Frame: 36 months ]
  • Effects on body composition measured with bioimpedance [ Time Frame: 36 months ]
  • Effects on haemoglobin (Hb), leucocyte and thrombocyte counts, FSH, LH, estradiol, Testosterone, inhibin B, TSH and Thyroxine [ Time Frame: 36 months ]
  • Muscle and fat mass measured by abdominal and mid-femoral computerized tomography [ Time Frame: 36 months ]
  • Activity of daily living measured a.m. Guralnik [ Time Frame: 36 months ]
  • Quality of life estimated by questionnaires [ Time Frame: 36 months ]


Original Secondary Outcome:

  • Bone mineral density measured by DXA
  • Effects on forced expiratory volume (Peakflow)
  • Standard photography appearance according to visual analogue scale (VAS)
  • Effects on free and total IGF-I, IGF-binding protein (BP)-1 and 3
  • Effects on lipids (fasting triglycerides(TG), total, HDL and LDL cholesterol)
  • Effects on body composition measured with bioimpedance
  • Effects on haemoglobin (Hb), leucocyte and thrombocyte counts, FSH, LH, estradiol, Testosterone, inhibin B, TSH and Thyroxine


Information By: Karolinska University Hospital

Dates:
Date Received: September 5, 2006
Date Started: April 2005
Date Completion:
Last Updated: September 11, 2016
Last Verified: September 2016